NCT02536755 (EXOSKEL) is a Phase 3 clinical trial of Eliglustat, GZ385660 in Gaucher Disease, sponsored by Genzyme, a Sanofi Company.

Who is sponsoring NCT02536755?

NCT02536755 is sponsored by Genzyme, a Sanofi Company.

What drugs are being tested in NCT02536755?

Interventions in NCT02536755: Eliglustat, GZ385660.

What condition does NCT02536755 study?

NCT02536755 studies Gaucher Disease.

Is NCT02536755 still recruiting?

NCT02536755 is currently completed. Last updated 15 July 2022.

Are results published for NCT02536755?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2022-07-15 · How we verify

NCT02536755: EXOSKEL

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Completed Phase 3 Results posted Last updated 15 July 2022

What this trial tests

Phase 3 trial testing Eliglustat, GZ385660 in Gaucher Disease in 31 participants. Completed in 24 June 2021.

Timeline

27 October 2015

Primary endpoint
24 June 2021

24 June 2021

Quick facts

Lead sponsor	Genzyme, a Sanofi Company
Phase	Phase 3
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	31
Start date	27 October 2015
Primary completion	24 June 2021
Estimated completion	24 June 2021
Sites	4 locations across Tunisia, Canada, Russia

Drugs / interventions tested

Eliglustat, GZ385660

Conditions studied

Gaucher Disease — all drugs for Gaucher Disease →

Sponsor

Genzyme, a Sanofi Company — full company profile →

Who can join

18 and older, any sex, with Gaucher Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

Mobility, i.e., ability to walk was assessed as a part of Gaucher disease assessment in participants. In this outcome measure, number of participants with their different mobility status along with the use of mobility aids (unrestricted mobility, walks with difficulty, walks with orthopaedic aid, requires wheelchair, bedridden) at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Unrestricted mobility: Study Baseline

Group	Value	95% CI
Eliglustat	31

Walks with difficulty: Study Baseline

Group	Value	95% CI
Eliglustat	0

Walks with orthopedic aid: Study Baseline

Group	Value	95% CI
Eliglustat	0

Requires wheelchair: Study Baseline

Group	Value	95% CI
Eliglustat	0

Bedridden: Study Baseline

Group	Value	95% CI
Eliglustat	0

Unrestricted mobility: Week 52

Group	Value	95% CI
Eliglustat	31

Walks with difficulty: Week 52

Group	Value	95% CI
Eliglustat	0

Walks with orthopedic aid: Week 52

Group	Value	95% CI
Eliglustat	0

Number of Participants With Bone Pain Levels During the Past 4 Weeks at Study Baseline, Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

Bone pain was assessed as a part of Gaucher disease assessment in participants. Participants were categorized as none (no bone pain), very mild bone pain, mild bone pain, moderate bone pain, severe bone pain and extreme bone pain during the past 4 weeks at each specified visit. In this outcome measure, number of participants with different level of bone pain during the past 4 weeks at specified time points were reported. Baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

None: Study Baseline

Group	Value	95% CI
Eliglustat	28

Very Mild: Study Baseline

Group	Value	95% CI
Eliglustat	1

Mild: Study Baseline

Group	Value	95% CI
Eliglustat	1

Moderate: Study Baseline

Group	Value	95% CI
Eliglustat	1

Severe: Study Baseline

Group	Value	95% CI
Eliglustat	0

Extreme: Study Baseline

Group	Value	95% CI
Eliglustat	0

None: Week 52

Group	Value	95% CI
Eliglustat	29

Very Mild: Week 52

Group	Value	95% CI
Eliglustat	1

Number of Participants With Bone Crisis at Study Baseline, Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

Bone crisis was assessed as a part of Gaucher disease assessment in participants. Acute, excruciating episodic bone pain is characteristic of Gaucher bone crisis, which typically causes periosteal elevation, elevated white blood cell count, fever, or debilitation lasting several days or longer and requires treatment with immobilization of the affected area, and opioid analgesics. Participants were categorized as 0= no bone crisis, 1= 1 bone crisis, 2= 2 bone crisis and \>=3 = more than 3 bone crisis during the assessment period. In this outcome measure, number of participants with different bo

Bone Crisis (0): Study Baseline

Group	Value	95% CI
Eliglustat	31

Bone Crisis (1): Study Baseline

Group	Value	95% CI
Eliglustat	0

Bone Crisis (2): Study Baseline

Group	Value	95% CI
Eliglustat	0

Bone Crisis (>=3): Study Baseline

Group	Value	95% CI
Eliglustat	0

Bone Crisis (0): Week 52

Group	Value	95% CI
Eliglustat	31

Bone Crisis (1): Week 52

Group	Value	95% CI
Eliglustat	0

Bone Crisis (2): Week 52

Group	Value	95% CI
Eliglustat	0

Bone Crisis (>=3): Week 52

Group	Value	95% CI
Eliglustat	0

Change From Current Study Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration. BMB score was measured using MRI (magnetic resonance imaging (MRI), ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the current study bas

Total BMB Score: Study Baseline

Group	Value	95% CI
Eliglustat	8.6667	± 3.3860

Total BMB Score: Week 52

Group	Value	95% CI
Eliglustat	8.5057	± 2.6482

Total BMB Score: Change at Week 52

Group	Value	95% CI
Eliglustat	-0.0256	± 1.6969

Total BMB Score: Week 104

Group	Value	95% CI
Eliglustat	9.1111	± 2.9627

Total BMB Score: Change at Week 104

Group	Value	95% CI
Eliglustat	1.2639	± 2.0897

Total BMB Score: Week 156

Group	Value	95% CI
Eliglustat	8.7821	± 2.6178

Total BMB Score: Change at Week 156

Group	Value	95% CI
Eliglustat	0.7101	± 1.8404

Total BMB Score: Week 208

Group	Value	95% CI
Eliglustat	8.9067	± 2.8844

Change From Eliglustat Baseline in Total Bone Marrow Burden (BMB) Scores at Weeks 52, 104, 156 and 208 Primary · Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

Bone marrow burden (BMB) scores indicate the degree of bone marrow infiltration was measured using MRI, ranged from 0 (no abnormalities) to 8 points (severe disease) for the lumbar spine and from 0 (no abnormalities) to 8 points (severe disease) for the femurs. The total BMB score was calculated as the sum of scores for femur and lumbar spine regions which ranged from 0 (no abnormalities) to 16 (severe disease) points. A higher BMB score signified more severe bone marrow involvement. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status

Total BMB Score: Eliglustat Baseline

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	10.4286	± 2.8146
Eliglustat: Participants From ENCORE	6.8333	± 2.4114

Total BMB Score: Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	8.4533	± 2.7502
Eliglustat: Participants From ENCORE	8.8333	± 2.1858

Total BMB Score: Change at Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-1.7179	± 2.0765
Eliglustat: Participants From ENCORE	2.0000	± 1.2472

Total BMB Score: Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	8.9420	± 3.1537
Eliglustat: Participants From ENCORE	10.0833	± 1.2874

Total BMB Score: Change at Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.1818	± 2.0459
Eliglustat: Participants From ENCORE	3.2500	± 2.6580

Total BMB Score: Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	8.6250	± 2.6600
Eliglustat: Participants From ENCORE	10.6667	± 0.9428

Total BMB Score: Change at Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-1.2424	± 2.3098
Eliglustat: Participants From ENCORE	3.3333	± 2.3570

Total BMB Score: Week 208

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	8.7879	± 3.0334
Eliglustat: Participants From ENCORE	9.7778	± 1.3878

Change From Current Study Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

Bone Mineral Density (BMD) measurements of the spine and bilateral femur were acquired by dual energy X-Ray absorptiometry (DXA) scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at Baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Total Spine BMD: Study Baseline

Group	Value	95% CI
Eliglustat	1.1449	± 0.1236

Total Spine BMD: Week 52

Group	Value	95% CI
Eliglustat	1.1476	± 0.1255

Total Spine BMD: Change at Week 52

Group	Value	95% CI
Eliglustat	-0.0029	± 0.0282

Total Spine BMD: Week 104

Group	Value	95% CI
Eliglustat	1.1326	± 0.1217

Total Spine BMD: Change at Week 104

Group	Value	95% CI
Eliglustat	-0.0055	± 0.0333

Total Spine BMD: Week 156

Group	Value	95% CI
Eliglustat	1.1373	± 0.1173

Total Spine BMD: Change at Week 156

Group	Value	95% CI
Eliglustat	-0.0007	± 0.0484

Total Spine BMD: Week 208

Group	Value	95% CI
Eliglustat	1.1236	± 0.1146

Change From Eliglustat Baseline in Total Spine and Femur Bone Mineral Density (BMD) at Weeks 52, 104, 156 and 208 Primary · Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

BMD measurements of the spine and bilateral femur were acquired by DXA scan. Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Total Spine BMD: Eliglustat Baseline

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	1.0757	± 0.1322
Eliglustat: Participants From ENCORE	1.1350	± 0.0896

Total Spine BMD: Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	1.1495	± 0.1307
Eliglustat: Participants From ENCORE	1.1245	± 0.0035

Total Spine BMD: Change at Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.0582	± 0.0656
Eliglustat: Participants From ENCORE	0.0195	± 0.0530

Total Spine BMD: Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	1.1280	± 0.1216
Eliglustat: Participants From ENCORE	1.1615	± 0.1371

Total Spine BMD: Change at Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.0600	± 0.0633
Eliglustat: Participants From ENCORE	0.0265	± 0.0617

Total Spine BMD: Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	1.1354	± 0.1153
Eliglustat: Participants From ENCORE	1.1498	± 0.1480

Total Spine BMD: Change at Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.0712	± 0.0718
Eliglustat: Participants From ENCORE	0.0148	± 0.0692

Total Spine BMD: Week 208

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	1.1168	± 0.1120
Eliglustat: Participants From ENCORE	1.1757	± 0.1466

Change From Current Study Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Total Spine T-score: Study Baseline

Group	Value	95% CI
Eliglustat	-0.4413	± 0.9782

Total Spine T-score: Week 52

Group	Value	95% CI
Eliglustat	-0.4362	± 1.0155

Total Spine T-score: Change at Week 52

Group	Value	95% CI
Eliglustat	-0.0250	± 0.2356

Total Spine T-score: Week 104

Group	Value	95% CI
Eliglustat	-0.5566	± 0.9522

Total Spine T-score: Change at Week 104

Group	Value	95% CI
Eliglustat	-0.0479	± 0.2751

Total Spine T-score: Week 156

Group	Value	95% CI
Eliglustat	-0.5152	± 0.9075

Total Spine T-score: Change at Week 156

Group	Value	95% CI
Eliglustat	-0.0066	± 0.4013

Total Spine T-score: Week 208

Group	Value	95% CI
Eliglustat	-0.6238	± 0.9015

Change From Eliglustat Baseline in Spine and Femur Total T-Scores for BMD at Weeks 52, 104, 156 and 208 Primary · Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The T-score bone density categories were: normal (score \>-1), osteopenia (score -2.5 to \<=-1), and osteoporosis (score \<= -2.5). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Total Spine T-score: Eliglustat Baseline

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.9381	± 1.0443
Eliglustat: Participants From ENCORE	-0.3000	± 0.8485

Total Spine T-score: Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.4063	± 1.0530
Eliglustat: Participants From ENCORE	-0.7950	± 0.0354

Total Spine T-score: Change at Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.5126	± 0.5622

Total Spine T-score: Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.5808	± 0.9512
Eliglustat: Participants From ENCORE	-0.4050	± 1.0898

Total Spine T-score: Change at Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.5215	± 0.5229
Eliglustat: Participants From ENCORE	0.5200	± 0.5657

Total Spine T-score: Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.5172	± 0.8875
Eliglustat: Participants From ENCORE	-0.5025	± 1.1771

Total Spine T-score: Change at Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.6150	± 0.6000
Eliglustat: Participants From ENCORE	0.4300	± 0.7495

Total Spine T-score: Week 208

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.6570	± 0.8823
Eliglustat: Participants From ENCORE	-0.3700	± 1.2194

Change From Current Study Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories were: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Total Spine Z-score: Study Baseline

Group	Value	95% CI
Eliglustat	-0.3687	± 0.8529

Total Spine Z-score: Week 52

Group	Value	95% CI
Eliglustat	-0.3338	± 0.8597

Total Spine Z-score: Change at Week 52

Group	Value	95% CI
Eliglustat	-0.0092	± 0.2405

Total Spine Z-score: Week 104

Group	Value	95% CI
Eliglustat	-0.4555	± 0.8522

Total Spine Z-score: Change at Week 104

Group	Value	95% CI
Eliglustat	-0.0245	± 0.2945

Total Spine Z-score: Week 156

Group	Value	95% CI
Eliglustat	-0.4034	± 0.8464

Total Spine Z-score: Change at Week 156

Group	Value	95% CI
Eliglustat	0.0276	± 0.4275

Total Spine Z-score: Week 208

Group	Value	95% CI
Eliglustat	-0.5035	± 0.8289

Change From Eliglustat Baseline in Spine and Femur Total Z-Scores for BMD at Weeks 52, 104, 156 and 208 Primary · Eliglustat Baseline, Weeks 52, 104, 156 and 208 of the current study

BMD measurements of the spine and bilateral femur were acquired by DXA scan. The Z-score bone density categories are: normal (score \>-2) and below normal (score \<=-2). Worst total femur at Baseline refers to the "worst" diseased left or right femur at baseline. For this outcome measure, baseline refers to the eliglustat baseline, which was defined as participant's status at the time of first dose of eliglustat in the previous Phase 2 or Phase 3 study.

Total Spine Z-score: Eliglustat Baseline

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.8826	± 0.9824
Eliglustat: Participants From ENCORE	-0.5750	± 0.6185

Total Spine Z-score: Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.2954	± 0.8851
Eliglustat: Participants From ENCORE	-0.7950	± 0.0354

Total Spine Z-score: Change at Week 52

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.5030	± 0.4230
Eliglustat: Participants From ENCORE	0.0550	± 0.3889

Total Spine Z-score: Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.4636	± 0.8357
Eliglustat: Participants From ENCORE	-0.4050	± 1.0898

Total Spine Z-score: Change at Week 104

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.5186	± 0.4578
Eliglustat: Participants From ENCORE	0.1700	± 0.5553

Total Spine Z-score: Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.3876	± 0.8129
Eliglustat: Participants From ENCORE	-0.5025	± 1.1771

Total Spine Z-score: Change at Week 156

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	0.6276	± 0.5671
Eliglustat: Participants From ENCORE	0.0725	± 0.6233

Total Spine Z-score: Week 208

Group	Value	95% CI
Eliglustat: Participants From Phase 2, ENGAGE and EDGE	-0.5209	± 0.8017
Eliglustat: Participants From ENCORE	-0.3700	± 1.2194

Total Number of New or Worsening Osteonecrosis Events for Spine and Femur at Study Baseline, Week 52, 104, 156 and 208 Primary · Study Baseline, Weeks 52, 104, 156 and 208

Osteonecrosis was assessed by bone MRI and X-Ray for spine and by MRI for femur. Total number of new or worsening osteonecrosis events among all the participants with corresponding assessment at specified time points were reported in this outcome measure. For this outcome measure, baseline refers to the current study baseline, which was defined as status at this study (EFC13781) entry.

Spine Osteonecrosis (MRI): Study Baseline

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (MRI): Week 52

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (MRI): Week 104

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (MRI): Week 156

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (MRI): Week 208

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (X-ray): Study Baseline

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (X-ray): Week 104

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Spine Osteonecrosis (X-ray): Week 208

Group	Value	95% CI
Eliglustat	0.0000	0.000 – 0.000

Adverse events — posted to ClinicalTrials.gov

Time frame: Time from the first administration of the IMP to the last administration of the IMP + 5 days (up to 4 years, or until commercial eliglustat was available to participants through reimbursement or through the compassionate use [expanded access] program). Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Eliglustat

Serious: 4/31 (13%)

Deaths: 0/31

Serious adverse events (5 terms)

Reaction	System	Eliglustat
Pulmonary Tuberculosis	Infections and infestations	—
Meniscus Injury	Injury, poisoning and procedural complications	—
Bone Pain	Musculoskeletal and connective tissue disorders	—
Ovarian Cyst	Reproductive system and breast disorders	—
Uterine Polyp	Reproductive system and breast disorders	—

Other adverse events (8 terms — click to expand)

Reaction	System	Eliglustat
Pregnancy	Pregnancy, puerperium and perinatal conditions	—
Iron Deficiency Anaemia	Blood and lymphatic system disorders	—
Toothache	Gastrointestinal disorders	—
Human Chorionic Gonadotropin Increased	Investigations	—
Arthralgia	Musculoskeletal and connective tissue disorders	—
Intervertebral Disc Protrusion	Musculoskeletal and connective tissue disorders	—
Osteopenia	Musculoskeletal and connective tissue disorders	—
Spinal Pain	Musculoskeletal and connective tissue disorders	—

Most-reported serious reactions: Pulmonary Tuberculosis, Meniscus Injury, Bone Pain, Ovarian Cyst, Uterine Polyp.

Data from ClinicalTrials.gov NCT02536755 adverse events section.

Sponsor's own description

Primary Objective: Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies. Secondary Objective: Evaluate the safety of eliglustat (by serious adverse event continuous monitoring), the quality of life (Short Form-36 Health Survey \[SF-36\]) and biomarkers of Gaucher disease type 1 (GD1) (chitotriosidase, plasma glucosylceramide \[GL-1\] and lyso glucosylceramide \[lyso-GL-1\]) in adult participants who successfully completed one of the Phase 2 or Phase 3 studies.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.

Verify or expand the search:

Other recruiting trials for Gaucher Disease

Currently open trials in the same condition.

NCT06528080 — A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease · EARLY_PHASE1 · active not recruiting
NCT06573723 — Institutional Registry of Rare Diseases · recruiting
NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases · recruiting
NCT05843552 — Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease · recruiting
NCT05487599 — A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) · Phase 1, PHASE2 · recruiting

Other Genzyme, a Sanofi Company trials

Trials by the same sponsor.

NCT06666413 — China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD · Phase 4 · recruiting
NCT05164055 — Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase) · Phase 4 · active not recruiting
NCT05134571 — China Post-marketing Surveillance (PMS) Study of Aldurazyme® · Phase 4 · completed
NCT05054387 — China Post-marketing Surveillance (PMS) Study of Fabrazyme® · Phase 4 · completed
NCT04676373 — Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen · Phase 4 · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02536755 (US National Library of Medicine, public domain)
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Genzyme, a Sanofi Company
Last refreshed: 15 July 2022

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02536755.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing