Trial record 1 of 1 for:    NCT01037309
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Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Prosensa Therapeutics
ClinicalTrials.gov Identifier:
NCT01037309
First received: December 21, 2009
Last updated: December 19, 2013
Last verified: December 2013
  Purpose

The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Drug: PRO044 SC
Drug: PRO044 IV
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by Prosensa Therapeutics:

Primary Outcome Measures:
  • To assess the dystrophin expression in the muscle biopsies by immunofluorescence analyses of cross-sections and by western blot analyses of total protein extracts [ Time Frame: Within 13 weeks after 5 weeks of treatment ] [ Designated as safety issue: No ]
  • Safety and tolerability of PRO044 [ Time Frame: During the 5 weeks of treatment and during the 13 weeks after treatment ] [ Designated as safety issue: Yes ]
  • Determine the pharmacokinetics of PRO044 [ Time Frame: During the 5 weeks of treatment and during the 13 weeks after treatment ] [ Designated as safety issue: No ]

Enrollment: 18
Study Start Date: December 2009
Study Completion Date: October 2013
Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PRO044, cohort 1
Subcutaneous injection of 0.5 mg/kg on day 1, 8, 15, 22 and 29.
Drug: PRO044 SC
Subcutaneous injection, once a week, for five weeks
Experimental: PRO044, cohort 2
Subcutaneous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29.
Drug: PRO044 SC
Subcutaneous injection, once a week, for five weeks
Experimental: PRO044, cohort 3
Subcutaneous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29.
Drug: PRO044 SC
Subcutaneous injection, once a week, for five weeks
Experimental: PRO044, cohort 4
Subcutaneous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29.
Drug: PRO044 SC
Subcutaneous injection, once a week, for five weeks
Experimental: PRO044, cohort 5
Subcutaneous injection of maximally 10 mg/kg on day 1, 8, 15, 22 and 29
Drug: PRO044 SC
Subcutaneous injection, once a week, for five weeks
Experimental: PRO044, cohort 6
Subcutaneous injection of maximally 12 mg/kg on day 1, 8, 15, 22 and 29
Drug: PRO044 SC
Subcutaneous injection, once a week, for five weeks
Experimental: PRO044, cohort 7
Intravenous injection of maximally 1.5 mg/kg on day 1, 8, 15, 22 and 29
Drug: PRO044 IV
Intravenous injection, once a week, for five weeks
Experimental: PRO044, cohort 8
Intravenous injection of maximally 5 mg/kg on day 1, 8, 15, 22 and 29
Drug: PRO044 IV
Intravenous injection, once a week, for five weeks
Experimental: PRO044, cohort 9
Intravenous injection of maximally 8 mg/kg on day 1, 8, 15, 22 and 29
Drug: PRO044 IV
Intravenous injection, once a week, for five weeks

Detailed Description:

To assess the effect of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To assess the safety and tolerability of PRO044 at different dose levels in subjects with Duchenne muscular dystrophy To determine the pharmacokinetics of PRO044 at different dose levels after subcutaneous and intravenous administration in subjects with Duchenne muscular dystrophy.

  Eligibility

Ages Eligible for Study:   5 Years to 16 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Boys aged between 5 and 16 years inclusive.
  2. Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO044.
  3. Life expectancy of at least 6 months.
  4. No previous treatment with investigational medicinal treatment within 6 months prior to the start of the (pre)-screening for the study.
  5. No previous treatment with idebenone within 6 months prior to the start of the (pre)-screening for the study.
  6. Willing and able to adhere to the study visit schedule and other protocol requirements.
  7. Written informed consent signed (by parent(s)/legal guardian and/or the patient, according to the local regulations).
  8. Glucocorticosteroids use which is stable for at least 2 months prior first drug administration.

Exclusion Criteria:

  1. Aberrant RNA splicing and/or aberrant response to PRO044, detected by in vitro PRO044 assay during pre-screening.
  2. Known presence of dystrophin in ≥ 5% of fibers in a pre-study diagnostic muscle biopsy.
  3. Severe muscle abnormalities defined as increased signal intensity in >50% of the tibialis anterior muscle at MRI.
  4. FEV1 and/or FVC < 60% of predicted.
  5. Current or history of liver or renal disease.
  6. Acute illness within 4 weeks prior to treatment (Day 1) which may interfere with the measurements.
  7. Severe mental retardation which in the opinion of the investigator prohibits participation in this study.
  8. Severe cardiac myopathy which in the opinion of the investigator prohibits participation in this study.
  9. Need for mechanical ventilation.
  10. Creatinine concentration above 1.5 times the upper limit of normal (age corrected).
  11. Serum ASAT and/or ALAT concentration(s) which suggest hepatic impairment.
  12. Use of anticoagulants, antithrombotics or antiplatelet agents.
  13. Use of idebenone.
  14. Use of any investigational product within 6 months prior to the start of the (pre)-screening for the study.
  15. Subject has donated blood less than 90 days before the start of the (pre)-screening for the study.
  16. Current or history of drug and/or alcohol abuse.
  17. Participation in another trial with an investigational product.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01037309

Locations
Belgium
UZ Leuven
Leuven, Belgium, 3000
Italy
S.Anna Hospital
Ferrara, Italy
Netherlands
Leiden University Medical Center
Leiden, Netherlands, 2300
Sweden
The Queen Silvia Children's Hospital
Gothenburg, Sweden
Sponsors and Collaborators
Prosensa Therapeutics
Investigators
Principal Investigator: A. Ferlini, PhD Università di Ferrara and S.Anna Hospital, Ferrara, Italy
Principal Investigator: J. J.G.M. Verschuuren, MD Leiden University Medical Center, Leiden, the Netherlands
Principal Investigator: N. Goemans, MD UZ Leuven, Leuven, Belgium
Principal Investigator: M. Tulinius, MD The Queen Silvia Children's Hospital, Gothenburg, Sweden
  More Information

No publications provided

Responsible Party: Prosensa Therapeutics
ClinicalTrials.gov Identifier: NCT01037309     History of Changes
Other Study ID Numbers: PRO044-CLIN-01
Study First Received: December 21, 2009
Last Updated: December 19, 2013
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
Italy: National Institute of Health
Netherlands: Ministry of Health, Welfare and Sport
Sweden: Medical Products Agency

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on September 22, 2014